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| Name | Class |
|---|---|
| Peking Union Medical College Hospital | OTHER |
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The goal of this clinical trial is to evaluate the safety and efficacy of IVB102 injection in subjects with XLRS.
This is a open label, dose escalation, single-center study. One eye of each participant will receive a single IVB102 injection by intravitreal injection. Participants will be followed for 52 weeks after which they will continue to be followed for up to 5 years after enrollment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| IVB102 Treatment Arm(Low dose) | Experimental | Intraocular injection of a single low dose of IVB102 |
|
| IVB102 Treatment Arm(Intermediate dose) | Experimental | Intraocular injection of a single intermediate dose of IVB102 |
|
| IVB102 Treatment Arm(High dose) | Experimental | Intraocular injection of a single high dose of IVB102 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IVB102 Injection | Genetic | Gene transfer by intravitreal injection of the RS1 AAV vector |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of DLTs | Number and proportion of dose limited toxicity(DLTs) | 4 weeks |
| Incidence of AEs | Number and severity of overall and ocular Adverse Events (AEs) | 52 weeks |
| Incidence of SAEs | Number and severity of overall and ocular Serious Adverse Events (SAEs) | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Visual function | Change from baseline in BCVA(Best Corrected Visual Acuity)(ETDRS) | 52 weeks |
| OCT imaging | Change from baseline in CST(Central Retinal Thickness)(OCT) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Beijing | Beijing Municipality | 100142 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 15937075 | Background | Pimenides D, George ND, Yates JR, Bradshaw K, Roberts SA, Moore AT, Trump D. X-linked retinoschisis: clinical phenotype and RS1 genotype in 86 UK patients. J Med Genet. 2005 Jun;42(6):e35. doi: 10.1136/jmg.2004.029769. | |
| 36212125 | Background | Wang XF, Chen FF, Zhou X, Cheng XX, Xie ZG. A novel mutation in RS1 and clinical manifestations in a Chinese twin family with congenital retinoschisis. Front Genet. 2022 Sep 23;13:993157. doi: 10.3389/fgene.2022.993157. eCollection 2022. |
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| ID | Term |
|---|---|
| D041441 | Retinoschisis |
| D012164 | Retinal Diseases |
| D012162 | Retinal Degeneration |
| D005128 | Eye Diseases |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
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| IVB102 Injection | Genetic | Gene transfer by intravitreal injection of the RS1 AAV vector |
|
| IVB102 Injection | Genetic | Gene transfer by intravitreal injection of the RS1 AAV vector |
|
| 52 weeks |
| ERG parameters | Change from baseline in response amplitudes measured using Electroretinogram (ERG) | 52 weeks |
| 17172462 | Background | Sikkink SK, Biswas S, Parry NR, Stanga PE, Trump D. X-linked retinoschisis: an update. J Med Genet. 2007 Apr;44(4):225-32. doi: 10.1136/jmg.2006.047340. Epub 2006 Dec 15. |